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Expert Interview

Slingshot members are talking to an expert! The topic is:

Exploring Breakthroughs in NF1-PN Treatment: Insights from SpringWorks' Phase 2b ReNeu Trial of Mirdametinib

Ticker(s): SWTX

Who's the expert?

Institution: Top Care Medical

  • Practicing pediatrician since 2017. In outpatient pediatrics and likes to focus on preventative care
  • Currently has 6 NF-1 patients in her clinic
  • Familiar with the recent update on the ReNeu study presented at ASCO

Interview Questions
Q1.

Can you elaborate on the significance of the 41% and 52% objective response rates in adults and children, respectively, from the Phase 2b ReNeu trial? How do these results compare to existing treatments for NF1-PN?

Added By: catalin_admin
Q2.

The ReNeu trial reported a median best change in tumor volume from baseline of -41% in adults and -42% in children. How does this deep and sustained reduction in tumor volume translate into clinical benefits for patients?

Added By: catalin_admin
Q3.

The trial showed significant improvements in patient-reported pain and health-related quality of life. How critical are these findings for the overall management and daily lives of NF1-PN patients?

Added By: catalin_admin
Q4.

With most adverse events being Grade 1 or 2, what can you tell us about the safety and tolerability of mirdametinib? How do these findings support the long-term use of this treatment in both adults and children?

Added By: catalin_admin
Q5.

The median time to onset of response was around 7.8 to 7.9 months, with the median duration of response not reached. How does this response time and duration impact the treatment regimen and expectations for patients?

Added By: catalin_admin
Q6.

Dermatitis acneiform, diarrhea, and nausea were among the most frequently reported treatment-related adverse events. What strategies can be employed to manage these side effects and ensure patient adherence to the treatment?


Added By: catalin_admin
Q7.

The ReNeu trial utilized blinded independent central review to confirm target tumor response. How does this methodological approach enhance the reliability and robustness of the trial results?

Added By: catalin_admin
Q8.

Given the compelling evidence from the ReNeu trial, what are the next steps for the approval and commercialization of mirdametinib? How soon can we expect this treatment to become available for NF1-PN patients?

Added By: catalin_admin

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